Clinical Research Directory

Data Collection of Standard Care and Evaluation of NHLBI Patients and Donors

Background: Researchers seek ways to study people s medical problems in order to teach and further general knowledge. The ability to assess and treat people with a wide range of diseases is critical to training people to be good doctors. It is also needed to keep medical staff up to date. In this study, researchers want to study the course of some illnesses to learn more about them. To do this, they will collect and review people s medical records. In some cases, they may also provide treatment. Objective: To collect data that may be used to help researchers create ideas for future research. Eligibility: People age 2 and older who have or are suspected to have a medical condition for which they have been referred to NIH s National Heart, Lung, and Blood Institute, as well as stem cell donors Design: Participants may be screened with a review of the following: Medical records Scans and images Other existing samples and reports. Participants medical data will be collected from the standard care they receive. This includes their routine blood and urine tests, X-rays and scans, and other tests to diagnose or follow their medical condition. Data will also be collected from the treatments they may receive. For stem cell donors, data from apheresis procedures will be collected. Demographic data will also be collected. All of the data will be kept in the medical records or on secure network drives. Some participants may need to be treated for their medical condition. If so, they will sign a separate consent form for that treatment. Participation lasts up to 2 years.

Prospective Multicenter Study of Blood mNGS for Diagnosing Invasive Pulmonary Fungal Disease in Hematologic Patients

The goal of this clinical trial is to learn whether a blood test called metagenomic next-generation sequencing (mNGS) can help diagnose invasive pulmonary fungal disease in patients with blood disorders. It will also evaluate how accurate this test is compared to traditional methods. The main questions it aims to answer are: Can blood mNGS accurately identify the fungi causing lung infections? How well does blood mNGS perform compared to conventional tests (such as culture, serum markers, and imaging)? Does the mNGS result influence doctors' decisions to start, change, or stop antifungal treatment? This study is a multicenter, prospective, observational trial. Researchers will compare the mNGS test with standard diagnostic methods to assess its usefulness in early diagnosis of fungal lung infections. Participants will: Have a blood sample collected within 72 hours of enrollment for mNGS testing Undergo routine clinical tests, including imaging, serum markers, and cultures, as part of standard care Be followed for 42 days to collect information on treatment and clinical outcomes

A Long-term Follow-up Study in Participants Who Received CTX001

This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161 (NCT05477563) (transfusion-dependent β-thalassemia \[TDT\] studies) or Study 121 (NCT03745287) or 151 (NCT05329649) or 161(NCT05477563) (severe sickle cell disease \[SCD\] studies).

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.

Clonal Hematopoiesis of Immunological Significance

Ambispective, national, multicenter observational cohort study aimed at characterizing the satellite dysimmune manifestations of clonal hematopoiesis, including Vexas (Vacuoles, E1 enzyme, X-linked, Autoinflammatory and Somatic) syndrome.

Pacritinib With Aza for Upfront Myelodysplastic Syndrome

This study will be conducted as a phase 1/2 study of safety and preliminary efficacy of pacritinib in combination with azacitidine for IPSS-M moderate low to very high risk MDS. Phase one will be a 3 + 3 design to assess the dose for the phase two portion. The phase two portion will employ a simon min-max two-stage design whereby fifteen patients will be enrolled in the first stage then ten more if at least two patients in stage one have a response. The dosing of pacritinib for the phase two study will be based on the phase one findings. Standard dosing of azacitidine will be used. A correlative study will be conducted in conjunction with the trial where the investigators will measure whole blood collected pre-treatment and at four days post-treatment to measure intracellular flow and phosflow to detect JAK/STAT, NF-κβ, and AKT/mTOR signaling in patient samples and how treatment affects these pathways.

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).

Gulf Long-Term Follow-Up Study

Background: * There has been little research on the long-term health effects from oil spills, even though at least 10 percent of all oil tanker spills between 1970 and 2009 have affected coastal populations. The Deepwater Horizon disaster, with its release of approximately 5 million barrels (\~680,000 tons) of crude oil into the Gulf of Mexico, is far larger than any of the individual tanker spills. Given the magnitude of this spill and the scope of the potential exposures, including the 55,000 workers involved in clean-up efforts and countless residents of the affected areas, researchers are interested in monitoring Gulf clean-up workers to understand the adverse consequences of oil spills in general. * The Gulf Long-term Follow-up Study will investigate health effects associated with the clean-up activities following the Deepwater Horizon disaster in the Gulf of Mexico on April 20, 2010. More than 100,000 persons completed safety training in preparation for participation in clean-up activities related to the spill. Many of these individuals participated in active clean-up efforts, but others did not. Exposures among persons involved in clean-up range from negligible to potentially significant, especially for workers involved in tasks associated with direct exposure to crude or burning oil, or to chemical dispersants. However, prediction of adverse health effects is not possible because the long-term human health consequences of oil spills are largely unknown. In addition to the oil itself, the widespread economic and lifestyle disruption caused by the oil spill may contribute to mental health problems among this population. Objectives: \- To investigate potential short- and long-term health effects associated with clean-up activities and exposures surrounding the Deepwater Horizon oil spill. Eligibility: \- English-, Spanish-, and Vietnamese-speaking workers and volunteers at least 21 years of age engaged or potentially engaged in oil spill clean-up operations in the Gulf of Mexico, or who lived in affected areas (Louisiana, Mississippi, Alabama, and Florida coastal regions). Design: * Participants will be divided into groups of those who performed oil-spill clean-up-related work ( exposed ) and those who did not engage in clean-up-related work ( unexposed controls). * Participants will be screened with a full medical history and physical examination, as well as an interview to determine the nature of their potential exposure. * Participants will provide blood, hair, toenail, urine, and saliva (spit) samples. Participants may also have a lung function exam. * Participants will have researchers collect dust from their homes by using wipes and special vacuum bags. * Participants will also provide detailed contact information, including their Social Security number, to be contacted in the future for long-term health follow-up appointments. These appointments will include 30-minute telephone interviews every 2 years.

Vorinostat for Graft vs Host Disease Prevention in Children, Adolescents and Young Adults Undergoing Allogeneic Blood and Marrow Transplantation

The purpose of this study is to determine the recommended phase 2 dose of the drug Vorinostat in children, adolescents and young adults following allogeneic blood or marrow transplant (BMT) and determine whether the addition of Vorinostat to the standard graft versus host disease (GVHD) prophylaxis will reduce the incidence of GVHD.

Autologous Transplantation of Human Cryopreserved Testis Tissue

For pre-pubertal boys undergoing gonadotoxic therapies, freezing immature testicular tissue (ITT) containing spermatogonial stem cells (SSCs) is currently the only option to potentially preserve future fertility. This experimental clinical study aims to provide proof-of-concept that frozen-thawed, ectopically autotransplanted adult human testicular tissue can support spermatogenesis in healthy adult men.

Advancing Health Information Exchange (HIE) During Inter-hospital Transfer (IHT) to Improve Patient Outcomes

Sub-optimal transfer of clinical information during inter-hospital transfer (IHT, the transfer of patients between acute care hospitals) is common and can lead to patient harm. To address this problem, the investigators will use key stakeholder input to refine and implement an interoperable health information exchange platform that integrates with the electronic health record and improves the reliability of and access to necessary clinical information in three use cases involving transfer of patients between sending and receiving hospitals with varying levels of affiliation and health record integration. The investigators will assess the effect of this intervention on frequency of medical errors, evaluate the use and usability of this platform from the perspective of those that interact with it, and use these results to develop a dissemination plan to spread implementation and use of this platform across other similar institutions.

Stem Cell Transplant From Donors After Alpha Beta Cell Depletion in Children and Adults With T-allo10 Cells Addback

The purpose of this study is to determine the safety of a cell therapy, T-allo10, after αβdepleted-HSCT in the hopes that it will boost the adaptive immune reconstitution of the patient while sparing the risk of developing severe Graft-versus-Host Disease (GvHD). The primary objective of Phase 1a is to determine the recommended Phase 2 dose (RP2D) administered after infusion of αβdepleted-HSCT in children and young adults with hematologic malignancies. A Phase 1b extension will occur after dose escalation, enrolling at the RP2D for the T-allo10 cells determined in the Phase 1 portion to evaluate the safety and efficacy of infusion of T-allo10 after receipt of αβdepleted-HSCT. Additionally, Phase 1b aims to explore improvements in immune reconstitution. All participants on this study must be enrolled on another study: NCT04249830

Bone Marrow Grafting for Leukemia and Lymphoma

The purpose of this study is to obtain tissue samples for ongoing studies regarding transplant outcomes and complications.

IL-5 CAR-T Cell Therapy for Refractory/Relapsed Eosinophilic Leukemia

This is an open-label, single-arm clinical study designed to evaluate the efficacy and safety of IL-5 CAR-T cell therapy in the treatment of patients with CD125-positive eosinophilic leukemia.

Muscle Dysfunction in Patients With Hematological Diseases Referred to Stem Cell Transplant

PURPOSE: To investigate the effect of the disease and HSCT on muscle dysfunction and to investigate the prognostic role of muscle dysfunction at critical decision points in patients with hematological diseases referred to hematopoietic stem cell transplant (HSCT). HSCT: Patients diagnosed with malignant hematological diseases who are referred to myeloablative HSCT, to a myeloablative "reduced toxicity conditioning" regime with Fludarabine and Treosulfane (FluTreo) or to non-myeloablative HSCT.

HEME Home Transfusion Program

This research study is evaluating whether a new care delivery program that provides access to home blood transfusions in hospice (i.e, HEME-Hospice) compared to regular standard of care improves quality of life, mood, and end-of-life health care utilization for patients with hematologic malignancies.

CD7 CAR-T Bridging to alloHSCT for R/R CD7+Malignant Hematologic Diseases

This is a single-arm, open-label, single-center, phase I/II study. The primary objective is to evaluate the safety of CD7 CAR-T Bridging to allo-HSCT therapy for patients with CD7-positive relapsed or refractory Malignant Hematologic Diseases

Muscle Dysfunction in Patients With Haematological Diseases

PURPOSE: To evaluate the prevalence and prognostic value of sarcopenia in patients diagnosed with hematological cancer diseases.

Visual and Auditory Distraction for the Relief of Pain and Anxiety

Bone marrow (BM) examinations are performed in diagnostic workup of malignancies, in particular hematological diseases, and are frequently associated with significant pain, distress, and anxiety. Analgesic and sedative medications are given to alleviate discomfort. The aim of this project is to study the effects of visual and auditory distraction on pain and anxiety during BM examinations. The project includes two baseline studies assessing pain perception, anxiety, and nociceptive response in patients and healthy volunteers undergoing BM examinations, and two randomized controlled trials evaluating the efficacy of VR distraction in reducing pain perception, anxiety, and nociceptive response. The intervention groups will use VR headsets, while control groups will follow standard procedure. Data collection include measurements of skin conductance, pulse, validated questionnaires and qualitative interviews. The project is strongly anchored in clinical practices and is a collaboration between Skåne University Hospital and Lund University, Sweden. The project group consists of three experts with relevant clinical and research competences. The project is ongoing, data collection will be finalized in 2028, and analyses and publications in 2027-2029. If distraction with VR results in less painful examinations and shows to be feasible in clinical praxis, the findings may contribute to development of new guidelines for pain management in BM-examinations and other painful medical procedures.

The Implementation of the Go Wish Game to Promote Advance Care Planning in Onco- Hematologic Disease

This is a mixed-method, device-free and drug-free multicenter interventional study. The study aims at facilitating end-of-life conversations within the doctor-patient relationship through the use of the Go Wish Game (GWG) and supporting patients, their caregivers and healthcare professionals to complete Advance Care Panning documentation. The GWG helps people clarify and identify their priorities, should they be affected by a chronic, disabling and potentially non-healing illness. In fact, the GWG consists of a small deck of cards, and on each card is a concrete action or situation that may be important to a person at the end of life. The "Onco-hema Go wish-ACP" project aims to evaluate the feasibility of a Go Wish Game-based intervention with patients with refractory lymphoma, leukemia or multiple myeloma or advanced solid tumors with prognosis \> 3 months. In terms of secondary objectives, the study aims to. * Evaluate and compare the intervention with hematology and oncology patients in terms of: - Other feasibility indicators; Involvement in CCP pathways; Quality of communication; Meaning of life; Impact on hope; through a series of questionnaires administered to patients and caregivers involved in the intervention * Qualitatively assess the acceptability of the intervention in terms of recruitment and delivery with patients and caregivers through semi-structured interviews and with professionals through Focus Groups (FGs). * To analyze the clinical records of enrolled patients in terms of: values and preferences; awareness of prognosis; end-of-life choices and shared decision-making on treatment decisions.

Stem Cell Transplant From Donors After Alpha Beta Cell Depletion in Children and Young Adults

The purpose of the CliniMACS® TCRαβ-Biotin System and CliniMACS® CD19 is to improve the safety and efficacy of allogeneic HLA-partially matched related or unrelated donors HSCT when no matched donors are available, to treat malignant and nonmalignant disorders for which HSCT is the recommended best available therapy. Initially this device will be used in a single-center, open-label, single-arm, phase II clinical trial to evaluate the efficacy of haploidentical PBSC grafts depleted of TCRα/β+ and CD19+ cells using the CliniMACS® TCRαβ/CD19 System in children and adults with hematological and non-hematological malignancies.

The Use of Residual De-identified Specimens and/or Samples From Patients for Clinical Research

Acquire residual human specimens and/or samples from patient samples which were sent for testing at LabPMM, LLC for Clinical Research

Continuous Wireless Monitoring of Vital Signs and Automated Alerts in Participants at Home and During Hospitalization

The primary aim of this study is to test and assess the implementation and effectiveness of continuous wireless vital signs monitoring with real-time alerts on: The frequency of patients monitored with adequate data quality as adequate clinical user satisfaction in the initial versus the last part of the trial (primary outcome).

Bleeding Events Before vs After Lowering Departmental Platelet Transfusion Trigger

Central venous catheters are essential when administering treatment for hematological conditions. Many patients have a decreased platelet count which increases the risk for bleeding complications. Baarle et al. recently published a randomized controlled study where withholding prophylactic platelet transfusions before CVC placement in patients with a platelet count of 10,000 to 50,000 per cubic millimeter did not meet the predefined margin for non-inferiority for postprocedural bleeding events (PMID: 37224197). However, bleedings grade 2 (defined as bleeding that requires external compression) were included despite lacking clinical significance. The aim of the present study is to investigate whether lowering the preprocedural platelet transfusion trigger from 50x10\^9/L to 10x10\^9/L for insertions of central venous catheters remains safe with regards to postprocedural bleeding events of grade 3-4.